Ponatinib (Iclusig) – GREAT NEWS!!!

I love news like this, especially when it’s been four-years in the making.

Iclusig (ponatinib) for the treatment of Chronic Myeloid Leukaemia (CML) in adult patients with chronic phase, accelerated phase, or blast phase CML who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

For CML patient in England, this means we now have another safety net that is readily available for consultants to prescribe immediately. This is going to make a big difference to treatment options and the mental well-being of many patients.

Just a quick shout to all of the people who work for and are associated with Incyte (formally ARIAD) who’ve never given up on this and have worked so hard to get it to us. Congratulations and thank you.

NICE has also recommended ponatinib for treating Philadelphia-chromosome-positive acute lymphoblastic leukaemia in adults. Hit the link for more info on this: www.leukaemiacare.org.uk/news/NICE-recommends-ponatinib

The full press release follows.

Kris

 

NICE Issues Positive Final Recommendation for Iclusig (ponatinib) for Chronic Myeloid Leukaemia (CML) in England

CML patients across the UK who are resistant or intolerant to second generation tyrosine kinase inhibitor (TKI) therapies will now have equal access to Iclusig

LONDON, UK [28 April 2017] – Incyte Corporation (Nasdaq:INCY) announces that the National Institute for Health and Care Excellence (NICE) Technology Appraisal Committee (TAC) has published a positive Final Appraisal Determination (FAD) recommending Iclusig^â (ponatinib) for the treatment of Chronic Myeloid Leukaemia (CML) in adult patients with chronic phase, accelerated phase, or blast phase CML who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.[i]

The positive FAD from NICE brings CML patients in England in line with those in Wales and Scotland who have had full-access to Iclusig, according to its license,[ii]^,[iii] since 2015; providing patients with CML across the UK who have failed other treatments equal access to an additional and important option.

“Today’s decision is important for patients with CML who have failed other treatments, as well as for physicians in England, who will now have access to the clinical benefits of Iclusig,” commented Mark Tanner, General Manager of Incyte Bioscience UK. “Together with the CML community, we have worked very hard over the last four years to encourage NICE to reconsider their original evaluation and are delighted that NICE has acknowledged the unmet need and the value that Iclusig brings.”

CML is a rare blood cancer with around 700 new cases each year in the UK.[iv]  CML affects economically active people, with around 50 percent of UK cases in people aged under 65 years.^iv Many patients with a new diagnosis of CML have a prolonged clinical benefit from targeted therapy with tyrosine kinase inhibitors (TKIs). However, there has been a high unmet need and poor prognosis for patients whose advanced disease is resistant and intolerant to other therapies.[v] Once available treatment options are exhausted, the prognosis can be poor.^v  Despite advances in treatment, there remains a need for additional effective therapies for the management of CML.[vi] Iclusig fulfils an important need in the treatment pathway for CML patients and provides clinicians and patients with a full suite of treatment options for CML.

Professor Jane Apperley, Department of Haematology, Imperial College School of Medicine, Hammersmith Hospital, London said, “This is an exciting and long-awaited outcome, which allows physicians to manage patients in a logical and clinical-evidence based manner with the goals of improving long-term survival and providing a good quality of life.”

Iclusig was approved by the European Commission[vii] in 2013 as an orphan drug for the treatment of adults with chronic phase, accelerated phase, or blast phase CML who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate. In Ph+ALL (Philadelphia chromosome‒positive Acute Lymphoblastic Leukaemia) patients, Iclusig is licensed for adult patients with Ph+ ALL who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate. Iclusig is also licensed for people with CML and PH+ALL who have T315I mutation.[viii]

About CML and Ph+ ALL

CML is a cancer of the white blood cells that is diagnosed in approximately 7,000 patients each year in Europe.[ix] CML is characterized by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality that produces the BCR-ABL protein. After a chronic phase of production of too many white blood cells, CML typically evolves to the more aggressive phases referred to as accelerated phase and blast crisis. Ph+ ALL is a subtype of acute lymphoblastic leukaemia that carries the Ph+ chromosome that produces BCR-ABL. It has a more aggressive course than CML and is often treated with a combination of chemotherapy and tyrosine kinase inhibitors. The BCR-ABL protein is expressed in both of these diseases.

About Iclusig^® (ponatinib) tablets

Iclusig targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

In the EU, Iclusig is approved for the treatment of adult patients with chronic phase, accelerated phase or blast phase chronic myeloid leukemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation, or the treatment of adult patients with Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

Incyte has an exclusive license from ARIAD Pharmaceuticals, Inc, a wholly owned subsidiary of Takeda Pharmaceutical Company Limited, to develop and commercialize Iclusig in the European Union and 28 other countries, including Switzerland, Norway, Turkey, Israel and Russia.

About Incyte

Incyte Corporation is a U.S.-based biopharmaceutical company focused on the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit the Company’s website at www.incyte.com.

Follow @Incyte on Twitter at https://twitter.com/Incyte.

[i] NICE. 2017. Final Appraisal Determination: Ponatinib for treating chronic myeloid leukaemia and acute lymphoblastic leukaemia. Available at https://www.nice.org.uk/guidance/gid-ta10060/documents/final-appraisal-determination-document Last accessed 28 April 2017

[ii] All Wales Medicines Strategy Group. Ponatinib (Iclusig). Appraisals. Available at: http://www.awmsg.org/awmsgonline/app/appraisalinfo/1163. Last accessed 24 March 2017

[iii] Scottish Medicines Consortium. SMC Advice. Ponatinib (Iclusig). Available at: http://www.scottishmedicines.org.uk/SMC_Advice/Advice/1032_15_ponatinib_Iclusig/ponatinib_Iclusig. Last accessed April 2017.

[iv] CRUK. Chronic myeloid leukaemia (CML) incidence statistics. Available at: http://www.cancerresearchuk.org/cancer-info/cancerstats/types/leukaemia-cml/incidence/. Last accessed April 2017.

[v] Cortes JE, KimD-W, Pinilla-Ibarz J, et al. A Phase 2 Trial of Ponatinib in Philadelphia Chromosome–Positive Leukemias. N Engl J Med 2013;369: 1783-1796. Available at: http://www.nejm.org/doi/pdf/10.1056/NEJMoa1306494.

[vi] Woessner DW, Lim CS, Deininger MW. Development of an Effective Therapy for CML. Cancer J 2011;17(6):doi:10.1097/PPO.0b013e318237e5b7. Available at: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3251313/pdf/nihms-332259.pdf. Last accessed April 2017.

[vii] EMA. Iclusig EPAR summary for the public.  Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002695/human_med_001656.jsp&mid=WC0b01ac058001d124. Last accessed April 2017.

[viii] Iclusig Summary of Product Characteristics. Available at: http://www.medicines.org.uk/emc/medicine/28145. Last accessed April 2017.

[ix] Rohrbacher M, Hasford J. Epidemiology of chronic myeloid leukaemia (CML). Best Pract Res Clin Haematol. 2009 Sep;22(3):295-302. Based on current estimate of population of Europe (738,199,000 in 2010).

 

Bloodwise Video – Kris Griffin (me)

Long time no see. I hope all is well. Just wanted to drop in with some shameless self promotion and to celebrate my association with Bloodwise (formally Leukaemia and Lymphoma Research).

I recorded this video some time ago now, it’s just been put live. I’m delighted with the results and I’d urge you to have a look at this and the other videos to learn a little more about the lives of blood cancer patients.

Thanks, Kris

Trial: Discontinuation of dasatinib

The results of this trial represent an incredible leap forward for CML patients who, like me, are on dasatinib (sprycel). For the patients in this trial, nearly 50% who stopped dasatinib maintained a deep molecular response. The other 50% started taking tablets again and all regained a deep molecular response.

This represents a huge benefit for the patient who could, effectively, remain drug-free but it also represent an economic benefit. What was once considered an expensive drug could soon be considered a drug-for-life for only half of the patients who take it. This could be enough to present a new case to NHS England over funding.

Thanks, Kris

Taken from The Lancet haematology

Summary
Background
First-line imatinib treatment can be successfully discontinued in patients with chronic myeloid leukaemia after deep molecular response has been sustained for at least 2 years. We investigated the safety and efficacy of discontinuing second-line or subsequent dasatinib after at least 1 year of deep molecular response.

Methods
The Dasatinib Discontinuation trial was a prospective multicentre trial done in Japan. Eligible patients taking dasatinib and with confirmed stable deep molecular response were enrolled between April 1, 2011, and March 31, 2012. All patients received dasatinib consolidation therapy for at least 1 year. In those with sustained deep molecular response, dasatinib was discontinued. Patients were followed up every month in year 1 (clinical cutoff), every 3 months in year 2, and every 6 months in year 3 for deep molecular response and immunological profiles. The primary endpoint was the proportion of patients with treatment-free remission at 6 months after discontinuation. Molecular relapse was defined as loss of deep molecular response at any assessment. This study is registered, number UMIN000005130.

Findings
88 patients were enrolled in the consolidation phase, 24 were excluded from the discontinuation phase due to fluctuations in BCR-ABL1 transcript levels. One patient was excluded because of positive expression of major and minor BCR-ABL1 transcripts in chronic myeloid leukaemia cells and the detection of minor BCR-ABL1 transcripts during consolidation. Thus, 63 patients discontinued dasatinib treatment. The 25 patients who were excluded from discontinuation continued to receive dasatinib and none showed disease progression. Median follow-up was 20·0 months (IQR 16·5–24·0). Of the 63 patients who discontinued and were not excluded, 30 patients maintained deep molecular response while 33 patients had molecular relapses, all within the first 7 months after discontinuation. The estimated overall treatment-free remission was 49% (95% CI 36–61) at 6 months. No severe treatment-related toxic effects were seen. Treatment was restarted in the 33 patients with relapse; rapid molecular responses were seen in all 33 patients, of whom 29 (88%) regained deep molecular response within 3 months, as did the remaining four by 6 months.

Interpretation
Dasatinib discontinuation after sustained deep molecular response for more than 1 year is feasible.

Funding
Epidemiological and Clinical Research Information Network (ECRIN).

Cancer Drugs Fund Cuts CML Drugs (and what we can do about it)

Details from the recent announcement:

The Cancer Drugs Fund in England will no longer pay for 16 medicines, used in 23 separate cancer treatments.
All the drugs on the Cancer Drugs Fund list have been rejected by the NHS as a whole because they do not provide enough benefit for the amount they cost.
At the beginning of 2015, there were 84 funded therapies, but after a series of culls there are now just 41.
The fund was set up by Prime Minister David Cameron to provide access to such medication. However, NHS England announced that the fund was due to go £100m over budget in 2014-15.
The drugs will be formally removed on 4 November and the announcement will not affect patients currently receiving treatment through the fund.
Patients affected: Blood cancer – 1,759 patients.
The Rarer Cancers Foundation said the news was a “hammer blow” and estimated that 5,500 patients across a spectrum of cancers would miss out.

Source: Cancer drugs fund cuts 23 treatments from BBC News.
The official announcement from NHS England can be found here.

Before I start it is imperative to start that the announcement will not affect patients currently receiving treatment through the fund.

I’ve read a lot of things over the last few days. I’ve heard many opinions and chewed a lot of fat. Any way you look at this recent decision, it’s hard to take any positives from it; that’s clearly why emotions are running so high. But, let’s remember what Yoda taught us:

“Fear is the path to the dark side. Fear leads to anger. Anger leads to hate. Hate leads to suffering.“

This is not a situation for finger-pointing or blaming people, countries or administrations we perceive to be at fault or guilty for a variety of suspected sins. One of the silliest suggestions I’ve read is that if that if we weren’t talking so many refugees in to the UK we would be able to afford the CML drugs. Not the case. Health economics doesn’t work like this. I’m not a fan of the Trident programme but I’m not daft enough to think that by scrapping it and saving billions we’d immediately be rewarded with the drugs we need. No, it’s more likely we’d get another station for High Speed 2. Joke. And for the record, I’m in favour of the UK playing our part and taking refugees.

We stand alone on this, fight our corner strategically and productively and make sure our voices are heard. Do I believe that campaigning hard will result in a reversal of this decision? No. But if we allow our voices to fall silent, when the day comes to start appraising drugs again, I want CML drugs to be at the front of people’s minds. I want people to understand that this is a poor decision about drugs that SAVE LIVES. I want the people responsible for the decisions to know that we are NOT faceless. I want them to know our names.

So what should we do? I believe there are two fundamental priorities to focus on:

1. To pressure the health administration groups in England to review decisions, open the appraisal process and ensure that we are part of the process moving forward – with respect to the reconfigured way of deciding which drugs to approve and which to reject.

2. To encourage pharmaceutical companies who manufacture our drugs to reduce their prices through Patient Access Schemes (PAS).

By playing this straight down the middle we position ourselves as the result of both health administration AND pharmaceutical company decisions. The decisions are unfair and unjust but that argument won’t win us any battles. A coordinated, strategic approach will. This means responding to requests for help with media enquiries, visiting Parliament to talk to MPs and writing letters to appropriate parties. It also means making yourself a more informed patient, understanding the process and contributing towards any changes. This is the only way we, as patients, will be part of any changes.

We’re doing this for our generation and the generation of patients that follow us. We’re doing this for the person diagnosed tomorrow who currently has fewer drugs available to them than when I was diagnosed 8 years ago. If that isn’t motivation enough to bring about change then I don’t know what is.

Thanks, Kris

Ponatinib: Scotland 1, Wales 1, England 0

In recent news I reported that Iclusig® (ponatinib) had been granted access to patients in Wales by NHS Wales. You can read the whole story here. I’m pleased to be able to write today to say that patients in Scotland have been given access to ponatinib too! Both countries are providing access for ALL phases of chronic myeloid leukaemia (CML).

In England ponatinib is only available on the Cancer Drugs Fund (CDF) IF the patient has the T315i mutation. We recently reported the majority of patients were being turned down in England after making individual funding requests; full story here.

Whilst you can read the full details of the appraisal by NHS Scotland I’ll draw your attention to this:

A non-comparative phase II study of ponatinib was conducted with primary outcomes of major cytogenetic response in patients with baseline chronic phase CML and major haematologic response in patients with baseline accelerated or blast phase CML or Ph+ALL. Ponatinib demonstrated efficacy in heavily pre-treated CML and Ph+ALL patients who had received dasatinib/nilotinib as second line or further line tyrosine kinase inhibitor therapy or who had the T315I mutation.

The studies of ponatinib show it to be very effective and NHS Scotland have recognised this, but NHS England remain steadfast and refuse to appraise ponatinib because the patient numbers are too low. We find ourselves in a situation where patients aren’t able to access a drug that could save their lives. How can the system be fair when, depending on which NHS authority you come under, will depend on the availability of a drug to you?

There is little that can be done at the moment but once Parliament reconvenes we’ll start to apply pressure and ask direct questions of the organisations that are allowing us to fall behind our counterparts in the United Kingdom.

Thanks, Kris

Advice: following a full submission considered under the orphan and end of life process:

ponatinib (Iclusig®) is accepted for use within NHS Scotland.

Indication under review: Adult patients with
• Chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.
• Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

A non-comparative phase II study of ponatinib was conducted with primary outcomes of major cytogenetic response in patients with baseline chronic phase CML and major haematologic response in patients with baseline accelerated or blast phase CML or Ph+ALL. Ponatinib demonstrated efficacy in heavily pre-treated CML and Ph+ALL patients who had received dasatinib/nilotinib as second line or further line tyrosine kinase inhibitor therapy or who had the T315I mutation.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.

Taken from: www.scottishmedicines.org.uk/SMC_Advice/Advice/1032_15_ponatinib_Iclusig/ponatinib_Iclusig

Ponatinib: Wales 1 England 0

After the bad news yesterday, where it was announced that 25 cancer drugs will be removed from the Cancer Drugs Fund (read the full story here), I come with better news. NHS Wales will be providing access to ponatinib for ALL phases of chronic myeloid leukaemia. In England ponatinib is only available on the Cancer Drugs Fund if the patient has the T315i mutation. This progressive step by NHS Wales, based on the Phase 2 PACE trial, gives patients in Wales another, much needed line of treatment against CML. I hope that authorities in England, Scotland and Northern Ireland take note of this step forward when they assess, or reassess, ponatinib for their respective countries.

Thanks, Kris
—

The positive assessment from the AWMSG is the first UK Health Technology Assessment (HTA) of Iclusig

Leatherhead, UK, 9 January 2015 — The Minister for Health and Social Services in Wales has ratified the recommendation from the All Wales Medicines Strategy Group (AWMSG) to approve Iclusig® (ponatinib) as a cost-effective treatment to be used in NHS Wales for the treatment of all phases of chronic myeloid leukaemia (CML) and Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL), in accordance with Iclusig’s licensed indication.

CML is a cancer of the white blood cells that is diagnosed in approximately 7,000 patients each year in Europe. The incidence of CML in Wales is 1.4 and 0.9 per 100,000 males and females, respectively. Iclusig is a targeted cancer medicine discovered and developed at ARIAD Pharmaceuticals, Inc.

“We are delighted that the Minister has endorsed the positive AWMSG recommendation, recognising the innovative nature of Iclusig and the potential benefit it can bring to cancer patients in Wales,” said Mark Tanner, General Manager, ARIAD UK. “Our goal is to deliver innovative solutions that address gaps in care for patients who are left with few clinical treatment options. Iclusig offers a new treatment option for many of these patients.”

The approval by the AWMSG was based on results from the pivotal Phase 2 PACE (Ponatinib Ph+ ALL and CML Evaluation) trial in patients with CML or Ph+ ALL who were resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy, or who had the T315I mutation of BCR-ABL. In Europe, Iclusig was approved in July 2013 for the treatment of adult patients with:

• Chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation;
• Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation.

“We welcome the ratification of the recommendation from the AWMSG for the treatment of CML and Ph+ ALL with Iclusig in all its licensed indications,” commented David Ryner, Chair of the Chronic Myeloid Leukaemia Support Group. “Although the number of patients qualifying for treatment will be limited, access to Iclusig represents an opportunity to make a significant difference to their lives. We would like to see the same opportunity made available to all other qualifying patients, no matter where they live in the UK.”

About Iclusig® (ponatinib)
Iclusig is a kinase inhibitor. The primary target for Iclusig is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Iclusig was designed using ARIAD’s computational and structure-based drug design platform specifically to inhibit the activity of BCR-ABL. Iclusig targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment, including the T315I mutation, which has been associated with resistance to other approved TKIs.

About CML and Ph+ ALL
CML is a cancer of the white blood cells that is diagnosed in approximately 7,000 patients each year in Europe.Error: Reference source not found CML is characterised by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality that produces the BCR-ABL protein. After a chronic phase of production of too many white blood cells, CML typically evolves to the more aggressive phases referred to as accelerated phase and blast crisis. Ph+ ALL is a subtype of acute lymphoblastic leukaemia that carries the Ph+ chromosome that produces BCR-ABL. It has a more aggressive course than CML and is often treated with a combination of chemotherapy and tyrosine kinase inhibitors. The BCR-ABL protein is expressed in both of these diseases.

About ARIAD
ARIAD Pharmaceuticals, Inc., headquartered in Cambridge, Massachusetts and Lausanne, Switzerland, is an integrated global oncology company focused on transforming the lives of cancer patients with breakthrough medicines.  ARIAD is working on new medicines to advance the treatment of various forms of chronic and acute leukaemia, lung cancer and other difficult-to-treat cancers.  ARIAD utilises computational and structural approaches to design small-molecule drugs that overcome resistance to existing cancer medicines.  For additional information, visit http://www.ariad.com or follow ARIAD on Twitter (@ARIADPharm).

Iclusig® is a registered trademark of ARIAD Pharmaceuticals, Inc.

You can download the original release here: AWMSG approval_FINAL release_090115

World CML Day 2014

Early next year it’ll be 7 years since I was diagnosed with Chronic Myeloid Leukaemia. I was 32.

Today is World CML Day. The story goes that, for no reason, two of my chromosomes had broken, swapped over and created a new, naughty, one called the Philadelphia Chromosome. This sent rubbish message to my body and could have easily killed me . My white blood count was 192 on diagnosis. It should have been 7.

The (nearly) 7 intervening years have been life changing. I grew up a lot. Got married. Had a son. Fund-raised. Lobbied government. Met great people. Got better. And became a better person.

For me and a small amount of people it’s CML day every day. There are only 650 of us newly diagnosed in the UK every year. But the breakthroughs being made in the CML world have implications on cancer treatment everywhere.

I’m treated daily with two small white tablet called dasatinib. They’ve put me back together. Two small white tablets are treating my cancer. All being well I should have normal life expectancy. My hopes and dreams, outside of Luca playing for Norwich one day, are that all cancers can be treated and cured with two small white tablets. Imagine that. That’s why I keep working, volunteering and supporting, we’ve achieved so much but there is still a long way to go.

Thanks to everyone who has helped me on my journey. I’ll finish with three photo, possibly the three biggest moments in my life – all three happened after diagnosis. Have a great day everyone. K.

Wedding Day

Luca was born

Major molecular response to CML

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#‎worldcmlday‬ ‪#‎leukemia‬

CML HORIZONS 2014 DAY 3

Late nights and early mornings. A snatched breakfast and sat, politely and on time, for the final session. There’s no rest at CML Horizons, we are straight into STOP trails. Normally spoken in hushed tones by patients, this is closest we get to a cure for CML on TKI therapy. It involves stopping therapy, only with the support of a clinician, once the disease is completely under control.

Giuseppe Saglio took us through the many forms of STOP trials which are mainly undertaken when the patient has achieved a swift (within 24 months) complete molecular response CMR (4 or 4.5 log reduction). There are other factors: women do better and swift progress to CMR is key. Some STOP trials have shown fluctuation below 0.1% of patients who have stopped, but the disease never goes beyond this point – they never lose response. It shows that continual monitoring is a key part to managing this method of treatment. The strength of the TKI the patients takes has a great bearing on the success of stopping treatment, there is a better success rate on nilotinib and dasatinib than on imatinib. Professor Saglio suggested, based on trials, that STOP was not risky and no patient accelerated their condition if the trail did not work. Even multiple stopping strategy can be used, but carful planning undertaken before each try.

Professor Saglio, “discontinuation of drugs will become common clinical practice”.

I’ve highlighted that line, it shows how far we have come and it gives hope for CML patients everywhere.

Susan Saubele provided an overview of interferon therapy in STOP trials. More good news, in particular with people who haven’t received a deep response. One interferon study found only 25% of people lost remission when combined therapy was withdrawn. It appears that major molecular remission (MMR) in interferon therapy is a good place to be; Pegasys interferon therapy achieved a better response. The EURO-SKI trial is the largest and most exciting study currently running, worth watching results as they come in later this year.

Neil Shah presented an individual case study where a woman came off imatinib due to pregnancy in 2001. She didn’t start the therapy again and remains, in 2014, undetectable. She is monitored annually. An amazing circumstance. It’s clear that our understanding of the disease and associated stem cells is growing year-on-year. Results from a variety of different studies look promising. The issue here, for patients, is that the topic is weighed down with scientific content and heavy discussion. I have to take an editorial decision and simply tell you the outlook is very positive. If you want the detailed analysis I would highly recommend Neil Shah’s presentation on the CML Advocates website when Horizons goes live, digitally, shortly after the conference. In the meantime, the morning session brought great hope to the room and there are many people, all over the world, working hard to develop deeper responses for all patients and a TKI cure.

After a smashing book presentation by Mina and Stephene Daban from LMC France, Gail Sperling from The Leukemia & Society in the USA stepped up to present social work and managing distress. I’m a keen believer in mindfulness and I feel it is the area in advocacy where we are most lacking. Whilst clinical treatment is first class, psychological well-being isn’t. Gail spoke about the ‘CML Busters’ – a group set up to deal with information and supporting patients. A very successful initiative. We explored self care and self assessment, ensuring that patients are happy and fulfilled and are dealing with things. The importance of being physically, emotionally, psychologically and spiritually healthy (as much as possible) is key. It’s about finding a balance. I have reservations about the term ‘self’ care – no one should be alone and support is not a solo venture. Gail was reassuring that the focus of her group was ensuring people had people; the ‘buddy’ programme was particularly impressive. The secret to success? Persistence, sharing, staying connected and a sense of humour.

The final session of the conference was from Sarah Liptrott, a clinical research nurse from the European Institute of Oncology in Milan. This fascinating talk on sex, self-esteem and a chronic disease was sincere and interesting. I was particularly impressed by the analysis of side effects in relation to intimacy and sex…kissing with ‘dry mouth’. Very real problems. Fatigue is a key factor and Sarah provided some excellent advice on dealing with it: fluids, setting goals, sleep, routines, eating healthily and yoga. As we heard from Gail, encouraging patients to be proactive in their care helps with self-esteem. In a similar way our treatment is becoming more and more personalised, our care is moving this way too.

I am constantly irritated by studies with tiny (less than 10 respondents) that advocate men staying on treatment whilst trying for a baby. Why take the risk? Sperm bank before treatment starts! There is not enough long-term data that gives us the right to make that decision. This area needs much further discussion, with clear guidelines, supported by stronger data. I feel like I say that every year, perhaps I do.

A wonderful conference. This is the 12th CML Horizons conference and they really go from strength-to-strength, the content will be available digitally on the CML Advocates website in the next 2 weeks and I’d recommend going through the information. Great thanks go out to everyone involved in putting together these marvellous 3 days.

I’d also like to thank Leukaemia & Lymphoma Research (LLR) in the UK for supporting my trip. Victoria Goldsmith, from LLR, accompanied me and I believe the information we bring back and the contacts we made will have a huge impact, not just on CML patients but on ALL blood cancer patients. LLR have been conspicuous by their absence at CML Horizons since I first attended. I applaud the foresight and drive they show in supporting patients via new initiatives like this. Thank you to everyone at Leukaemia & Lymphoma Research, for everything you do.

Time to fly…via Amsteram…wishing everyone a safe journey now and forever.

CML HORIZONS 2014 DAY 2

On a normal Saturday morning I’d be getting up early with my little boy, Luca, and waiting for him to calm down (normally takes 4-5 hours) watching football and taking it easy. It’s an honour to change that routine this weekend and at 9am I am sat in a conference in Belgrade, Serbia with over 200 delegates; patients and families, clinicians and representatives from organisations who all have a vested interest in Chronic Myeloid Leukaemia (CML).

Our first session covered CML in real life – comorbidities and drug interaction. An outstanding review from Dr Neil Shah provided an overview of age and side effects. It was fascinating to hear the prescriptive differences that come with different age groups, essentially trying to balance tolerance and side effects. There is obviously a strong call for proper management of risks and benefits. I was also interested in the ease at which Dr Shah would move from one treatment to another, clearly not a problem when drugs are available and paid for. But, very much, the perfect scenario.

CML Horizons does a brilliant job in balancing the sessions and Professor Andrija Bogdanovic presented a localised example, from Serbia, of the situation he works in. It’s interesting to see him working with older drugs including hydroxyurea and achieving spectacular results, 78.6% complete cytogenetic response rate after 12 months! His frustrations were clear when talking about the limitations he faced when trying to fund care, “put the pressure on insurance”. Andrija works in a situation very different to Neil Shah but both remain committed, flexible and dedicated to saving lives with the resources they have in hand.

It’s critical to understand how individual our bodies are and how much what we eat affects our drug intake. Dr Annette Freidbank’s session on drug interactions provided a very specific journey into the many ways drug effectiveness can be changed. For example food increasing the effectiveness of nilotinib (Tasigna) by 82%

Once again we heard the warnings about grapefruit affecting the effectiveness of TKIs and we were all very thankful it wasn’t served at breakfast. Natural and herbal products may also cause interactions, it’s a key responsibility to be safe and informed. Therefore, the relationship we have with our doctor is critical, being able to ask advice, both Doctor and patient working together to make sure that the way the drugs are tolerable and ensure the drugs work. It’s a very topical, useful area of investigation.

After a short break we were treated to a presentation from Dr Martin Godfrey about using social media. Sadly some technical issues cut the presentation short but this approach is vital to patient organisations. Even in the 3 years I’ve been attending CML Horizons I’ve seen the conference grow digitally at an exponential rate. It’s certainly making our world much smaller and easier to connect and navigate, for a rare cancer like Chronic Myeloid Leukaemia this is imperative. Whilst this session was a little dry it did provide an overview, for beginners, as to the practical benefits of using social media.

The issues of trust, access, e-safety and misinformation are very, very real. It’s important that we don’t advocate a one-size-fits-all approach. We’re still finding our feet. I’ve seen some amazing results on Twitter and on Facebook, Nigel Deekes’ CML UK Facebook group in particular. Likewise, I’ve seen some horror stories where people have lost control of stories, access or information. Very sad. But, we mustn’t forget this can happen with any form of media, it’s just that social media is far more immediate…hence the fear factor. Only time and knowledge will overcome this.

The differentiated approach to advocacy strategy from Tamas Bereczky was very enlightening. Placing the expert patient at the heart of strategy and ensuring they work with academics, regulators and industry really works, as long as we can keep them motivated! I particularly enjoyed his approach to policy and scientific work and finding the balance between the two, maintaining credibility and increasing knowledge. Tamas is a skilled board member at the European AIDS Treatment Group (EATG) and they have become very good at advocacy work over a long period of time. There is much we can learn from them. The European HIV Testing Week initiative worked very well and was adopted widely as a community initiative.
The case study of Gilead’s Sofosbuvir, which is a life-saving treatment of HVC, was very relevant to the CML audience. The cost of the drug was restrictive and EATG took on a classic activism role to raise the issue. Perhaps we CML patients are too polite, perhaps we need to change?

Finally Tamas presented a very practical approach to advocacy work, I’d recommend looking his slides up when CML Horizons goes digital shortly after the conference. EATG have also created an advocacy manual on their website – www.eatg.org – which I am looking forward to reading.

On to the session many people were waiting for, a packed room for The New Realities: Generics and Copy Drugs in CML. Yoseph Caraco looked at how we assess generic drugs. If you are unfamiliar with the concept of generics, these are drugs where the patent expires and anyone can manufacture them and release them, typically with a 70-90% cost reduction. A huge cost reduction, surely a benefit? The issue is quality. A colleague described this as a classic song being covered by another artist, sometimes it’s still a great song…sometimes it isn’t so good.

Imatinib has a low penetration rate, around 30%, with CML patients worldwide. This is due to the high cost. We were shown that some generics only showed very slight underperformance compared to the parent drug, however it still presents a risk, I certainly wouldn’t want to be part of the 2% that could lose their response. I find it very concerning that we would switch for price alone. When clinical trials are undertaken and quality assurance is given the reassurance means everything to the patient.

Cheryl-Anne Simoneau from the CML Society of Canada took the baton and presented patients as informed decision makers. Canada has a very proactive generic market as the prices are capped to 18%-20% of branded drug prices. Cheryl advocates patient power, ensuring that PCR results are known, tracked and monitored very closely. It is vital that patients are aware of the prescription they are given and if anything changes they report it immediately – this includes packaging as well as any side-effects, “your CML journey is unique to you”. The society is looking to lead by being an promoting the example of an autonomous, informed, engaged patient.

Andrija Bogdanovic and Qian Jiang, both haematologists, discussed generics from their perspective. A variety of issues: no practical experience, different forms, not enough published data, lots of conflicting data in the media and bad experiences. It was noted that the Serbian solution was to switch all 220 CML patients to generic imatinib (Anzovip). Within 3 months 7 patients lost response, they were placed back on the previous drug and regained response. There was a slightly higher loss of response from newly diagnosed patients, after 12 months, who started on imatinib (Anzovip) (IRIS trail comparison). It’s clear that the Serbian haematologists are monitoring the situation very carefully and their clinical approach to generics is very commendable.

Qian Jiang’s focus was whether bio-equivalence is the same as clinical equivalence. She presented an overview of the situation in China where many generics are being released, a differentiation in provincial funding confused the matter even further. The practical advice followed Cheryl-Anne Simoneau’s patient power, ask questions, risk mitigate, wait for the right moment and clinical evidence before switching. She ended by stating that, “generics should not be recommended to patients without solid evidence of clinical equivalence in spite of bio equivalence”.

There are lots of questions being asked of generic drugs, there is no clear answer on whether they are good or bad. It’s about risk mitigation and from a patient perspective not taking a risk, this is life or death after all. Whilst I understand, I have an issue with this coming down to cost. Without clinical trials, which increase the cost, I fear we won’t get the answer we are looking for. For the patient who has no choice, there are no branded drugs available to them at all, generics present a very real opportunity to cling to life…

After a enjoyable night out with friends, I sit writing this blog, contemplating whether it’s worth giving up family time. Nothing is that important, but sometimes we have to make sacrifices. It’s been a good day and I have plenty to take home with me, there’s still a day left. Sunday is just around the corner.

CML HORIZONS 2014 DAY 1

It was a long journey and an early morning. Sure, some people have travelled further and probably got up earlier but, sat writing this, I feel the pain. It was good to finally arrive in Serbia for CML Horizons 2014, no thanks to Air France who tried to derail me at every step. 

I was sad to have missed the opening video from Dr. Brian Drucker and the tribute to John Goldman, that pesky journey has much to answer for. I look forward to seeing the video and tribute on the CML Advocates website. So, things kicked off with a trip around the world with advocacy sessions: Latin America, Africa & Middle East, Asia-Pacific, North America and  Europe. This whistle-stop tour featured a key delegate from each region who presented an overview and the main challenges they faced. I was particularly struck, from Greg Stephens’ presentation, by the huge increase in CML deaths in North America. Whilst Greg is working on trying to understand what is causing this disastrous trend, I’d take a bet on it coming down to finance. The same old story. Very disappointing. It’s even more disappointing coming on the back of the excellent Leukaemia & Lymphoma Research, Impact Day, in London where we heard about the fantastic progress being made in the world of blood cancer.

Jan Geissler did his best to redress the balance with a more healthy European outlook but as we move West to East across Europe the challenges become greater. More disparity. All of the regions are working hard to bring a fair distribution of drugs that ultimately save lives. 

To the first medical session of the conference; Dr Neil Shah updated the delegates with the latest available treatments. Whilst we touched upon various forms of treatment including stem cell transplants and interferon-alpha the focus was on TKIs. The long term IRIS trial (7-8 year follow up), which is sadly coming to an end, shows a 93% survival rate considering only CML deaths for patients on Imatinib. Dr Shah felt that Imatinib has outperformed all expectations. We were presented with data that shows nilotinib and dasatinib as superior treatments to imatinib. For example in trails the complete cytogenetic response rate by 12 months on dasatinib is 83%, for imatinib 72%. For major molecular remission rate by 48 months, 76% on nilotinib and 58% on imatinib. This isn’t to say that imatinib isn’t the wonder drug it was first heralded as, without it this stunning progress could never have been made; and for those patients it suits, it works incredibly well. 

Next up, a fascinating update on therapy recommendations from Prof Giuseppe Saglio from the University of Turin, who really drilled the data down for us and showed a very logical progression of drugs and optimum responses. It is very clear that new drugs have given our clinicians excellent ammunition in the treatment of CML.

Finally, Dr Qian Jiang from the Peking University People’s Hospital who spoke about the role of transplant in CML. Whilst it is still a very important part of treating CML, the number of operations has steeply declined since the advent of TKIs. It is however, fascinating to see continuous development and improvement in this key area.

After a short question and answer session the day broke and we all raced back to our rooms to prepare for dinner. It was wonderful to catch up with old friends and see many new faces. Most conversations started, “has it really been a year?”. 

The CML community is lucky to have such a vibrant, resilient and informed group of people. With hard work and good bonds the group have take an orphan disease and put it firmly on the blood cancer map. Better than that, they have shown the way. The group is keen to talk about ‘advocacy’ and ‘blood cancer’ rather than isolate itself by being singular. This is the key, the group understands that the progress being made here and now will affect cancers of all types for generations to come.

Today has really set the pace for a fantastic conference. Stay tuned…same CML time….same CML channel.